Lucerastat study for untreated men with Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study tests a new medicine called lucerastat to see how well it works and how safe it is. It is for adult men with Fabry disease who have not been treated before. The medicine may help reduce the buildup of fatty substances in cells and improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a man aged 18 or older.
- You must have a confirmed diagnosis of Fabry disease, based on low enzyme activity or a gene mutation.
- You have had at least one of these signs: nerve pain (often in hands or feet), certain eye changes (cornea verticillata), or small dark red spots on skin (angiokeratomas).
- You have not had any Fabry disease treatment (like enzyme therapy or chaperone therapy) in the past 6 months, or you have never been treated.
- Your kidney function must be good enough: an estimated filtered blood test (eGFR) of at least 45 mL/min per 1.73 m2.
- A blood marker for Fabry disease (lyso-Gb3) must be at least 20 ng/mL.
- If you have diabetes, it should be well controlled (HbA1c of 8% or less).
- No recent stroke, heart attack, or surgery within 6 months, and no severe heart failure within the last 3 months.
- You must not be taking certain medicines like amiodarone or hydroxychloroquine in the past 6 months, and you must not have had gene or cell therapy before.
- You must be able to stop blood thinners if needed and not have bleeding problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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